Longitudinal observational study of sporadic inclusion body myositis: Implications for clinical trials

Neuromuscular Disorders

Volumn 23, Issue 5, 2013, Pages 404-412

  Cortese, A ^a,b   Machado, P ^a,c   Morrow, J ^a   Dewar, L ^a   Hiscock, A ^a   Miller, A ^a   Brady, S ^d   Hilton Jones, D ^d   Parton, M ^a   Hanna, M G ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b UNIVERSITY OF PAVIA   (Italy)

^c CENTRO HOSPITALAR E UNIVERSITÁRIO DE COIMBRA   (Portugal)

^d JOHN RADCLIFFE HOSPITAL   (United Kingdom)

Author keywords

Clinical trial; IBM functional rating scale; Inclusion body myositis; Muscle disease; Myometry; Outcomes measures

Indexed keywords

IMMUNOGLOBULIN; IMMUNOSUPPRESSIVE AGENT; STEROID;

ADULT; AGED; ARTICLE; CLINICAL ASSESSMENT; CLINICAL EFFECTIVENESS; CLINICAL FEATURE; CONTROLLED STUDY; DISABILITY SEVERITY; DISEASE DURATION; DYSPHAGIA; FEMALE; FOLLOW UP; HUMAN; IBM FUNCTIONAL RATING SCALE; INCLUSION BODY MYOSITIS; LONGITUDINAL STUDY; MAJOR CLINICAL STUDY; MALE; MANUAL MUSCLE TESTING; MUSCLE EXAMINATION; MUSCLE STRENGTH; OBSERVATIONAL STUDY; ONSET AGE; ORTHOSIS; PHYSICAL DISABILITY; PRIORITY JOURNAL; PROGNOSIS; PROSPECTIVE STUDY; QUADRICEPS FEMORIS MUSCLE; QUANTITATIVE MUSCLE TESTING OF QUADRICEPS MUSCLE; SENSITIVITY ANALYSIS; SURVIVAL TIME; WALKING AID; WALKING DIFFICULTY; WHEELCHAIR;

ADULT; AGED; AGED, 80 AND OVER; CLINICAL TRIALS AS TOPIC; DISEASE PROGRESSION; FEMALE; HUMANS; LONGITUDINAL STUDIES; MALE; MIDDLE AGED; MUSCLE, SKELETAL; MYOSITIS, INCLUSION BODY; WALKING;

EID: 84876073632     PISSN: 09608966     EISSN: 18732364     Source Type: Journal    
DOI: 10.1016/j.nmd.2013.02.010     Document Type: Article

References (25)

1. Scott A.P., Laing N.G., Mastaglia F., et al. Recombination mapping of the susceptibility region for sporadic inclusion body myositis within the major histocompatibility complex. J Neuroimmunol 2011, 235:77-83.
2. Larue S., Maisonobe T., Benveniste O., et al. Distal muscle involvement in granulomatous myositis can mimic inclusion body myositis. J Neurol Neurosurg Psychiatry 2011, 82:674-677.
3. Needham M., Mastaglia F.L. Inclusion body myositis: current pathogenetic concepts and diagnostic and therapeutic approaches. Lancet Neurol 2007, 6:620-631.
4. Askanas V., Engel W.K. Sporadic inclusion-body myositis: conformational multifactorial ageing-related degenerative muscle disease associated with proteasomal and lysosomal inhibition, endoplasmic reticulum stress, and accumulation of amyloid-beta42 oligomers and phosphorylated tau. Presse Med 2011, 40:e219-e235.
5. Machado P., Miller A., Holton J., Hanna M. Sporadic inclusion body myositis: an unsolved mystery. Acta Reumatol Port 2009, 34:161-182.
6. Cox F.M., Titulaer M.J., Sont J.K., Wintzen A.R., Verschuuren J.J., Badrising U.A. A 12-year follow-up in sporadic inclusion body myositis: an end stage with major disabilities. Brain 2011, 134:3167-3175.
7. Rose M.R., McDermott M.P., Thornton C.A., Palenski C., Martens W.B., Griggs R.C. A prospective natural history study of inclusion body myositis: implications for clinical trials. Neurology 2001, 57:548-550.
8. Dalakas M.C., Rakocevic G., Schmidt J., et al. Effect of Alemtuzumab (CAMPATH 1-H) in patients with inclusion-body myositis. Brain 2009, 132:1536-1544.
9. Griggs R.C., Askanas V., DiMauro S., et al. Inclusion body myositis and myopathies. Ann Neurol 1995, 38:705-713.
10. Tawil R., Griggs R.C. Inclusion body myositis. Curr Opin Rheumatol 2002, 14:653-657.
11. Hilton-Jones D., Miller A., Parton M., Holton J., Sewry C., Hanna M.G. Inclusion body myositis: MRC centre for neuromuscular diseases, IBM workshop, London, 13 June 2008. Neuromuscul Disord 2010, 20:142-147.
12. Dalakas M.C., Sonies B., Dambrosia J., Sekul E., Cupler E., Sivakumar K. Treatment of inclusion-body myositis with IVIg: a double-blind, placebo-controlled study. Neurology 1997, 48:712-716.
13. Jackson C.E., Barohn R.J., Gronseth G., Pandya S., Herbelin L. Inclusion body myositis functional rating scale: a reliable and valid measure of disease severity. Muscle Nerve 2008, 37:473-476.
14. Knesevich J.W., Toro F.R., Morris J.C., LaBarge E. Aphasia, family history, and the longitudinal course of senile dementia of the Alzheimer type. Psychiatry Res 1985, 14:255-263.
15. Cohen J. Statistical power analysis for the behavioral sciences 1977, Academic Press Inc., New York.
16. Lotz B.P., Engel A.G., Nishino H., Stevens J.C., Litchy W.J. Inclusion body myositis. Observations in 40 patients. Brain 1989, 112(Pt. 3):727-747.
17. Lindberg C., Persson L.I., Bjorkander J., Oldfors A. Inclusion body myositis: clinical, morphological, physiological and laboratory findings in 18 cases. Acta Neurol Scand 1994, 89:123-131.
18. Amato A.A., Gronseth G.S., Jackson C.E., et al. Inclusion body myositis: clinical and pathological boundaries. Ann Neurol 1996, 40:581-586.
19. Badrising U.A., Maat-Schieman M.L., van Houwelingen J.C., et al. Inclusion body myositis. Clinical features and clinical course of the disease in 64 patients. J Neurol 2005, 252:1448-1454.
20. Felice K.J., North W.A. Inclusion body myositis in Connecticut: observations in 35 patients during an 8-year period. Medicine (Baltimore) 2001, 80:320-327.
21. Crosby C.A., Wehbe M.A., Mawr B. Hand strength: normative values. J Hand Surg Am 1994, 19:665-670.
22. Hayter S.M., Cook M.C. Updated assessment of the prevalence, spectrum and case definition of autoimmune disease. Autoimmun Rev 2012, 11:754-765.
23. Benveniste O., Guiguet M., Freebody J., et al. Long-term observational study of sporadic inclusion body myositis. Brain 2011, 134:3176-3184.
24. Lowes L.P., Alfano L., Viollet L., et al. Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis. Muscle Nerve 2012, 45:163-168.
25. Peng A., Koffman B.M., Malley J.D., Dalakas M.C. Disease progression in sporadic inclusion body myositis: observations in 78 patients. Neurology 2000, 55:296-298.